Analysis of RAB27A gene in griscelli syndrome type 2: novel mutations including a deletion hotspot

J Clin Immunol. 2008 Jul;28(4):384-9. doi: 10.1007/s10875-008-9192-5. Epub 2008 Mar 19.

Abstract

Introduction: Griscelli syndrome type 2 is an autosomal recessive disorder characterized by pigmentary dilution and occurrence of acute phases of hemophagocytosis. The disease is caused by mutations in RAB27A gene, coding a small GTPase involved in terminal phases of cytotoxic granule/melanosome exocytosis.

Materials and methods: We describe the result of mutation analysis among nine patients from seven non-related Persian families. We present four novel mutations including a deletion hot spot (514del 5).

Conclusion: This hot spot is flanked by "direct repeats" of nucleotides, which are previously shown to be associated with areas of recurrent small deletions.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Age of Onset
  • Albinism / genetics*
  • Albinism / physiopathology
  • Base Sequence
  • Child
  • Child, Preschool
  • DNA Mutational Analysis
  • Female
  • Humans
  • Immunologic Deficiency Syndromes / genetics*
  • Immunologic Deficiency Syndromes / physiopathology
  • Infant
  • Male
  • Polymerase Chain Reaction
  • Sequence Deletion / genetics*
  • Syndrome
  • rab GTP-Binding Proteins / genetics*
  • rab27 GTP-Binding Proteins

Substances

  • rab27 GTP-Binding Proteins
  • RAB27A protein, human
  • rab GTP-Binding Proteins